Full Press Release Details
ProQR s Drug Candidate QRX-411 for Usher Syndrome Receives Orphan Drug
Designation from FDA and EMA
LEIDEN, the Netherlands, July 5, 2017 - ProQR
Therapeutics N.V. (Nasdaq:PRQR) today announced that the company s investigational drug QRX-411 has received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) and European
Medicines Agency (EMA) for the treatment of retinitis pigmentosa, including Usher syndrome, the subtype targeted by QRX-411. Usher syndrome is the leading cause of combined deafness and blindness due to
genetic defects in the Usher gene.
ODD in the U.S. and European Union provides a special status for investigational drugs being developed for rare
diseases. The ODD programs offer development program tax benefits and a waiver of the NDA application user fee, as well as market exclusivity for up to seven years in the U.S., and ten years in the European Union following market approval.
We are pleased with the progress we have made to date with our novel RNA based therapeutic ophthalmology pipeline for patients suffering from genetic
eye diseases. Securing orphan drug designations from the FDA and EMA for QRX-411 is a milestone for the program and highlights the importance of addressing the unmet need of this debilitating disease,
said Daniel A. de Boer, CEO of ProQR, The severe genetic retinal diseases we are targeting do not have any available therapies, especially disease modifying therapies focused on restoring vision or impeding progression of the disease. We
believe our novel RNA oligonucleotide approach has the potential to make a meaningful impact in the lives of Usher syndrome patients and others with rare genetic eye diseases.
ProQR Therapeutics N.V. | Zernikedreef 9, 2333 CK Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com
Chief Development Strategy Officer, David M. Rodman, MD, notes, At ProQR we have a unique opportunity to
combine the flexibility of our oligonucleotide drug discovery platform with accelerated drug development strategies for rare diseases. Orphan drug designation is an important step in rapidly bringing transformational precision medicines to
patients with Usher syndrome and many other genetic causes of blindness in children and adults.
ProQR s growing ophthalmology portfolio
About Usher Syndrome
Usher syndrome is the leading cause
of combined deafness and blindness. Patients with this syndrome generally progress to a stage in which they have very limited central vision and moderate to severe deafness. To date, there are no treatments approved or products in clinical
development that treat the vision loss associated with the disease. Usher syndrome Type II is one of the most common forms of Usher syndrome and is caused by mutations in the USH2A gene.
QRX-411 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause
of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QRX-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA.
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such
as cystic fibrosis, Leber s congenital amaurosis Type 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.
FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often
indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to , may, plan,
potential, predict, project, should, will, would and similar expressions. Forward-looking statements are based on management s beliefs and assumptions and on information
available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QRX-411 and the clinical development and therapeutic
potential thereof, statements regarding orphan drug designation, including the intended benefits of such status, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in
our opthamology portfolio, and statements regarding our oligonucleotide drug discovery platform. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation,
risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with
the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these
forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.
Director, Investor Relations